Tech Transfer eNews Blog

Bristol U start-up develops groundbreaking gene therapy for kidney diseases


By Jesse Schwartz
Published: January 13th, 2021

A Bristol University start-up has developed a novel technique to treat Nephrotic Syndrome, a rare genetic disease that is known to cause kidney failure.

Purespring Therapeutics has tested its gene-replacement treatment on mice, finding that it “almost completely” eradicated the disease, according to the start-up’s founders. In response to their success, biotech investment firm Syncona invest gave Purespring £45 million (over $61.2M US) in funding to develop the treatment for commercialization.

“This technique has massive potential,” says Moin Saleem, professor at Bristol University and co-founder of Purespring. “We have an ambition to treat thousands of people in the UK in the short to medium term. If it goes well, it could extend to the most common causes of chronic kidney disease within 10 years. Such is the potential of this technology. “

The treatment delivers healthy genes to the glomerulus, where the first stage of kidney filtration takes place, through a harmless Adeno-associated virus (AAV). This is the first time scientists targeting kidney disease have been able to deliver the right genes to exactly the right cells.

“The challenge is to introduce genetic material that reverses or cures disease into the specific kidney cell type that is affected by that disease — and not into other cells where it might cause side effects,” says Saleem. “We have solved the problem of directing the AAV to the correct kidney cell type, called the podocyte, so this opens up the possibility of treating many kidney diseases using curative genetic therapy for the first time.”

Chris Hollowood, chief investment officer at Syncona, comments, “We look forward to building a world class company around this innovative science, in order to develop therapies with the potential to deliver dramatic impact for patients.”

Source: iNews

Posted under: Tech Transfer e-News

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